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All Times EDT

Friday, September 25
Fri, Sep 25, 3:30 PM - 4:45 PM
Virtual
Estimands: Application of the Framework in Answering the Question of Interest in Drug Development

Implementing the Estimand Framework: An FDA Perspective (301216)

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*Mallorie Fiero, FDA CDER Office of Biostatistics 

Keywords: Estimand

The estimand framework was introduced in the ICH E9 (R1) addendum, which aims to align the design, conduct, and statistical analysis plan with a study's clinical questions of interest. FDA CDER's Office of Biostatistics (OB) recently formed an estimand working group to promote appropriate use of the estimand and collaborate with clinicians to implement the estimand framework across review divisions. This talk will go over OB's approach to implementing the estimand framework with a focus on considerations in patient-focused drug development and oncology. Common intercurrent events in oncology such as tumor progression, use of subsequent anti-cancer therapies, crossover (i.e., switching from control to the experimental arm), and death, pose a unique challenge when interpreting endpoints for regulatory use. The estimand framework helps multi-disciplinary teams to carefully think about how to address these intercurrent events within the clinical context of the study. Ultimately, this framework can provide clarity in the questions that are being answered and has potential to improve communication during regulatory drug-development.